Intellia Therapeutics Presents Data On Redosing With In Vivo CRISPR Therapy; 55 Mg NTLA-2001 Dose Shows 90% Median Reduction In Serum TTR At Day 28; Well Tolerated Across All Patients; Potential Redosing Advantage For Future Therapies
Intellia Therapeutics Presents Data On Redosing With In Vivo CRISPR Therapy; 55 Mg NTLA-2001 Dose Shows 90% Median Reduction In Serum TTR At Day 28; Well Tolerated Across All Patients; Potential Redosing Advantage For Future Therapies
intellia therapeutics展示了關於體內CRISPR治療再劑量的數據；55毫克NTLA-2001劑量顯示在第28天時血清TTR中位數減少了90％；所有患者都能良好耐受；爲未來治療提供潛在再劑量優勢。
- First-ever clinical data demonstrating redosing with an investigational in vivo CRISPR-based therapy
- Follow-on dosing with a 55 mg dose of NTLA-2001 led to a 90% median reduction in serum TTR at day 28 in the three patients who previously received the lowest dose in the Phase 1 dose-escalation study
- 55 mg follow-on dose was well tolerated across all patients
- While redosing is not planned for the NTLA-2001 program in transthyretin (ATTR) amyloidosis, a redosing option could be an important advantage of Intellia's non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired
- 首次臨床數據證明，在體內CRISPR基因編輯療法的治療方案中再次施用的有效性
- 在第一階段劑量遞增研究中，使用55毫克的Ntla-2001劑量進行隨訪治療，三名之前接受最低劑量的患者在第28天時血清TTR減少了90％
- 所有患者均能很好地耐受接受55毫克的隨訪劑量。
- 雖然在轉甲狀腺素運輸蛋白（ATTR）澱粉樣變（amyloidosis）中，NTLA-2001計劃中並不打算再次給藥，但再次施用選項可能是Intellia非病毒、脂質納米顆粒（LNP）爲基礎的遞送平台在未來研究中尋求目標效應時的重要優勢。
譯文內容由第三人軟體翻譯。
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